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Orpheris has successfully advanced the lead product, OP-101, in part due to over $20 M in NIH funding provided to our founders in collaboration with Johns Hopkins University, Wayne State University, NIH and the Kennedy Krieger Institute.

After successful meetings with FDA (pre-IND & Type C), we have established a regulatory path for approval of OP-101 in ccALD patients not eligible for bone marrow transplant as our first indication. We plan to submit the IND by the end of this year and initiate a single Phase 1/2/3 trial for approval in this first indication (NDA in 2021). We believe the peak revenue from the first indication will be $300M worldwide. We are also exploring second indications for OP-101 including Parkinson's disease, Huntington's disease, and ALS, which are more prevalent conditions, and we believe that revenues from this second indication would exceed $800M in the US alone.